簡介
鐮狀細胞貧血症為遺傳性(先天性)的血液疾病,血液中的紅血球形狀異常。主要的前兆、症狀為貧血和發育遲緩、視野障礙、疼痛、頻繁感染感染疾病等。主要的治療方法,有抗生素和止痛藥、輸血、幹細胞移植等。
本報告提供全球各國的鐮形血球貧血症治療用的開發中產品的開發情形相關分析、產品開發與上市的最新趨勢,及臨床實驗各階段的產品一覽,再加上主要企業簡介、主要藥物概要、最新的產業趨勢等資訊。
目錄
簡介
鐮形血球貧血症概要
治療藥的開發
- 鐮形血球貧血症的開發中產品:概要
- 鐮形血球貧血症的開發中產品:比較分析
各企業開發中的鐮形血球貧血症治療藥
大學/研究機關研究中的鐮形血球貧血症治療藥
開發中產品的概要
- 後期階段的產品
- 臨床實驗階段的產品
- 初期階段的產品
- 不明確階段的產品
鐮形血球貧血症治療藥:開發中產品一覽(各企業)
鐮形血球貧血症治療藥:研究中的產品一覽(各大學/研究機關)
鐮形血球貧血症治療藥的開發企業
Before the financial crisis, dealer market-making held a powerful place in the nation’s biggest investment banks. The fresh-out-of-college traders that shuffled in and out of Manhattan bank buildings each day handled the banks' inventories of bonds worth hundreds of millions of dollars, which they used as starting capital to make their own trades, says Lawrence McDonald, a former Lehman Bros. corporate bond trader. The holdings, which were used to make markets for each type of corporate security, would fit into two categories, or books.
“The front book is the facilitation book. That’s the book for client liquidity. The back book is your prop position. That book was responsible for a lot of profits on the Street in 2007, and that today pretty much doesn’t exist,” said McDonald, who authored A Colossal Failure of Common Sense, a bestseller about the bank's demise.
The smaller amount that dealers hold on their books means they can’t unconditionally buy bonds that investors are trying to sell, especially in large blocks
- Johnson & Johnson
- 第一三共
- Sangamo BioSciences, Inc.
- Gamida Cell Ltd.
- Novartis AG
- Astellas Pharma Inc.
- Addex Therapeutics
- Mast Therapeutics, Inc.
- Alnylam Pharmaceuticals, Inc.
- Cellerant Therapeutics, Inc.
- Advinus Therapeutics Ltd.
- Prolong Pharmaceuticals
- SynZyme Technologies, LLC
- Erytech Pharma SA
- Archemix Corp.
- Morphogenesis, Inc.
- NKT Therapeutics, Inc.
- Acetylon Pharmaceuticals, Inc.
- Emmaus Medical, Inc.
- AesRx, LLC
- Errant Gene Therapeutics, LLC
- Dilaforette AB
- OrphageniX, Inc.
- ReveraGen BioPharma, Inc.
鐮形血球貧血症:治療藥的評估
- 單劑治療藥的情況
- 並用治療藥的情況
- 各標的
- 各作用機制
- 各投藥法
- 各分子類型
藥物簡介
麩醯胺酸
硫酸鎂
vepoloxamer
Aes-103
regadenoson
montelukast sodium+羥基尿素
LentiGlobin
NiCord
panobinosutatto
zileuton CR(調整放出)
sevuparin sodium
Sanguinate
tetrahydrouridine+decitabine
NKTT-120
CNTO-530
Enhoxy
Trichostatin A
鐮形血球貧血症用P-Selectin抑制用核酸適體
異常血色素症用ZFP轉錄因子
鐮形血球貧血症用DNA寡核苷酸
VBP-15
PNQ-103
鐮形血球貧血症用遺傳基因療法
ALN-TMP
Lentiviral-Mediated遺傳基因療法
AIC-6020
ACY-957
VitalHeme
EdX-17
AIC-2011
非惡性血液疾病用中央記憶T細胞
HbF誘導物質計劃
Minihepcidins
SCPF
小分子TNFR1-NAM計劃
MSKCC
GTx011
CLT-001
鐮形血球貧血症治療藥:開發中產品的最新趨勢
鐮形血球貧血症治療藥:暫停開發的產品
鐮形血球貧血症治療藥:開發中止的產品
鐮形血球貧血症相關產品的開發里程碑
附錄
圖表一覽
※ 本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。
目錄
Summary
Global Markets Direct's, 'Sickle Cell Disease - Pipeline Review, H2 2014', provides an overview of the Sickle Cell Disease's therapeutic pipeline.
This report provides comprehensive information on the therapeutic development for Sickle Cell Disease, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Sickle Cell Disease and special features on late-stage and discontinued projects.
Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.
The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.
Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.
Scope
- The report provides a snapshot of the global therapeutic landscape of Sickle Cell Disease
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in the therapeutics development for Sickle Cell Disease and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects
- A review of the Sickle Cell Disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Sickle Cell Disease pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products
Reasons to buy
- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Sickle Cell Disease
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Sickle Cell Disease pipeline depth and focus of Indication therapeutics
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Table of Contents
- Table of Contents
- List of Tables
- List of Figures
Introduction
- Global Markets Direct Report Coverage
Sickle Cell Disease Overview
Therapeutics Development
- Pipeline Products for Sickle Cell Disease - Overview
- Pipeline Products for Sickle Cell Disease - Comparative Analysis
Sickle Cell Disease - Therapeutics under Development by Companies
Sickle Cell Disease - Therapeutics under Investigation by Universities/Institutes
Sickle Cell Disease - Pipeline Products Glance
- Late Stage Products
- Clinical Stage Products
- Early Stage Products
Sickle Cell Disease - Products under Development by Companies
Sickle Cell Disease - Products under Investigation by Universities/Institutes
Sickle Cell Disease - Companies Involved in Therapeutics Development
- Acceleron Pharma, Inc.
- Acetylon Pharmaceuticals, Inc.
- Addex Therapeutics Ltd
- Advinus Therapeutics Ltd.
- AesRx, LLC.
- Alnylam Pharmaceuticals, Inc.
- Archemix Corp.
- Bluebird bio, Inc.
- Bristol-Myers Squibb Company
- Children's Hospital Boston
- Daiichi Sankyo Company, Limited
- Dilaforette AB
- Emmaus Medical, Inc.
- Errant Gene Therapeutics, LLC
- Erytech Pharma SA
- Gamida Cell Ltd.
- Gilead Sciences, Inc.
- Global Blood Therapeutics, Inc.
- Isis Pharmaceuticals, Inc.
- Johnson & Johnson
- Mast Therapeutics, Inc.
- Morphogenesis, Inc.
- NKT Therapeutics, Inc.
- Novartis AG
- OrphageniX, Inc.
- Pfizer Inc.
- Prolong Pharmaceuticals
- ReveraGen BioPharma, Inc.
- Sangamo BioSciences, Inc.
- Sanofi
- SynZyme Technologies, LLC
Sickle Cell Disease - Therapeutics Assessment
- Assessment by Monotherapy Products
- Assessment by Target
- Assessment by Mechanism of Action
- Assessment by Route of Administration
- Assessment by Molecule Type
Drug Profiles
- ACY-957 - Drug Profile
- ADX-85142 - Drug Profile
- ADX-94819 - Drug Profile
- Aes-103 - Drug Profile
- AIC-2011 - Drug Profile
- AIC-6020 - Drug Profile
- ALN-TMP - Drug Profile
- Antisense Oligonucleotide to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile
- apixaban - Drug Profile
- ARC-5690 - Drug Profile
- Cell Therapy for Sickle Cell Disease - Drug Profile
- CNTO-530 - Drug Profile
- DNA Oligonucleotides for Sickle Cell Anemia - Drug Profile
- Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile
- EdX-17 - Drug Profile
- Enhoxy - Drug Profile
- Gene Therapy for Hemoglobinopathies - Drug Profile
- Gene Therapy for Sickle Cell Anemia - Drug Profile
- Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile
- Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile
- Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile
- glutamine - Drug Profile
- GTX-011 - Drug Profile
- LentiGlobin - Drug Profile
- luspatercept - Drug Profile
- NiCord - Drug Profile
- NKTT-120 - Drug Profile
- panobinostat - Drug Profile
- PB-04 - Drug Profile
- PF-4447943 - Drug Profile
- plerixafor - Drug Profile
- PNQ-103 - Drug Profile
- prasugrel hydrochloride - Drug Profile
- regadenoson - Drug Profile
- RN-1 - Drug Profile
- Sanguinate - Drug Profile
- SCPF - Drug Profile
- sevuparin sodium - Drug Profile
- Small Molecule to Agonize A2A for Immunology, Hematological and Neurological Disorders - Drug Profile
- Small Molecules to Inhibit TNFR1 for Immunology, Gastrointestinal, Neurology and Hematological Disorders - Drug Profile
- Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease - Drug Profile
- Synthetic Peptides Hematological and Genetic Disorders - Drug Profile
- Trichostatin A - Drug Profile
- VBP-15 - Drug Profile
- vepoloxamer - Drug Profile
- VitalHeme - Drug Profile
Sickle Cell Disease - Recent Pipeline Updates
Sickle Cell Disease - Dormant Projects
Sickle Cell Disease - Discontinued Products
Sickle Cell Disease - Product Development Milestones
Featured News & Press Releases
Appendix
- Methodology
- Coverage
- Secondary Research
- Primary Research
- Expert Panel Validation
- Contact Us
- Disclaimer
List of Tables
- Number of Products under Development for Sickle Cell Disease, H2 2014
- Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H2 2014
- Number of Products under Development by Companies, H2 2014
- Number of Products under Investigation by Universities/Institutes, H2 2014
- Comparative Analysis by Late Stage Development, H2 2014
- Comparative Analysis by Clinical Stage Development, H2 2014
- Comparative Analysis by Early Stage Development, H2 2014
- Products under Development by Companies, H2 2014
- Products under Development by Companies, H2 2014 (Contd..1)
- Products under Development by Companies, H2 2014 (Contd..2)
- Products under Investigation by Universities/Institutes, H2 2014
- Sickle Cell Disease - Pipeline by Acceleron Pharma, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Addex Therapeutics Ltd, H2 2014
- Sickle Cell Disease - Pipeline by Advinus Therapeutics Ltd., H2 2014
- Sickle Cell Disease - Pipeline by AesRx, LLC., H2 2014
- Sickle Cell Disease - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Archemix Corp., H2 2014
- Sickle Cell Disease - Pipeline by Bluebird bio, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Company, H2 2014
- Sickle Cell Disease - Pipeline by Children's Hospital Boston, H2 2014
- Sickle Cell Disease - Pipeline by Daiichi Sankyo Company, Limited, H2 2014
- Sickle Cell Disease - Pipeline by Dilaforette AB, H2 2014
- Sickle Cell Disease - Pipeline by Emmaus Medical, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Errant Gene Therapeutics, LLC, H2 2014
- Sickle Cell Disease - Pipeline by Erytech Pharma SA, H2 2014
- Sickle Cell Disease - Pipeline by Gamida Cell Ltd., H2 2014
- Sickle Cell Disease - Pipeline by Gilead Sciences, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Global Blood Therapeutics, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Isis Pharmaceuticals, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Johnson & Johnson, H2 2014
- Sickle Cell Disease - Pipeline by Mast Therapeutics, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Morphogenesis, Inc., H2 2014
- Sickle Cell Disease - Pipeline by NKT Therapeutics, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Novartis AG, H2 2014
- Sickle Cell Disease - Pipeline by OrphageniX, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Pfizer Inc., H2 2014
- Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals, H2 2014
- Sickle Cell Disease - Pipeline by ReveraGen BioPharma, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Sangamo BioSciences, Inc., H2 2014
- Sickle Cell Disease - Pipeline by Sanofi, H2 2014
- Sickle Cell Disease - Pipeline by SynZyme Technologies, LLC, H2 2014
- Assessment by Monotherapy Products, H2 2014
- Number of Products by Stage and Target, H2 2014
- Number of Products by Stage and Mechanism of Action, H2 2014
- Number of Products by Stage and Route of Administration, H2 2014
- Number of Products by Stage and Molecule Type, H2 2014
- Sickle Cell Disease Therapeutics - Recent Pipeline Updates, H2 2014
- Sickle Cell Disease - Dormant Projects, H2 2014
- Sickle Cell Disease - Discontinued Products, H2 2014
List of Figures
- Number of Products under Development for Sickle Cell Disease, H2 2014
- Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H2 2014
- Number of Products under Development by Companies, H2 2014
- Number of Products under Investigation by Universities/Institutes, H2 2014
- Comparative Analysis by Clinical Stage Development, H2 2014
- Comparative Analysis by Early Stage Products, H2 2014
- Assessment by Monotherapy Products, H2 2014
- Number of Products by Top 10 Targets, H2 2014
- Number of Products by Stage and Top 10 Targets, H2 2014
- Number of Products by Top 10 Mechanism of Actions, H2 2014
- Number of Products by Stage and Top 10 Mechanism of Actions, H2 2014
- Number of Products by Top 10 Routes of Administration, H2 2014
- Number of Products by Stage and Top 10 Routes of Administration, H2 2014
- Number of Products by Top 10 Molecule Types, H2 2014
- Number of Products by Stage and Top 10 Molecule Types, H2 2014
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